supports further clinical trials with Trofinetide

October 13, 2015

Cincinnati, OH - October 12, 2015 - today announced that the Board of Directors has made a financial commitment, through the Research to Reality Campaign, to continue support for further clinical trials with trofinetide, a drug developed and manufactured by Neuren Pharmaceuticals.

Following a successful Phase 2 trial with trofinetide in adult women with Rett syndrome, and an encouraging meeting with the Food and Drug Administration, Neuren is moving forward with a Phase 2 tolerability trial for children with Rett syndrome. Concurrently, Neuren is working with the FDA to design a Phase 3 pivotal study with trofinetide as a treatment for Rett syndrome.

Rajat Shah, Chairman of's, commented " is proud to continue our partnership with Neuren. We are grateful for their work and commitment to the clinical trials of trofinetide. We are hopeful that these clinical trials will be successful."

There are no approved drug treatments for Rett, a devastating neurological disorder. These trial advancements are significant because they bring us closer to the first potential treatments, and the hope of improving quality of life for those suffering from Rett syndrome.

This is an optimistic time for the Rett syndrome community. Research efforts are rapidly advancing our understanding of the disorder and the first treatments are closer to a reality. Ultimately, it is hoped they will lead to a cure.'s Research to Reality Campaign is ensuring that funding is available to advance promising clinical trials like those for trofinetide. is committed to a full spectrum approach to Rett syndrome research by supporting clinical trials as well as basic, translational and neurohabilitation research to better understand, treat, and ultimately cure Rett syndrome.
ABOUT RETTSYNDROME.ORG, a 501(c)3 non-profit, is the most comprehensive organization dedicated to accelerating research of treatments and a cure for Rett syndrome and related disorders while providing information and resources to empower Rett families. As the world's leading private funder of Rett syndrome research, has funded over $34M in high-quality, peer-reviewed research grants and programs to date. The organization hosts the largest global gathering of Rett researchers and clinicians to establish research direction for the future. To learn more about our work and Rett syndrome, visit or call (800) 818-7388 (RETT).

International Rett Syndrome Foundation

Related Clinical Trials Articles from Brightsurf:

Nearly 1 in 5 cancer patients less likely to enroll in clinical trials during pandemic
A significant portion of cancer patients may be less likely to enroll in a clinical trial due to the ongoing coronavirus pandemic.

COVID-19 clinical trials lack diversity
Despite disproportionately higher rates of COVID-19 infection, hospitalization and death among people of color, minority groups are significantly underrepresented in COVID-19 clinical trials.

Why we should trust registered clinical trials
In a time when we have to rely on clinical trials for COVID-19 drugs and vaccines, a new study brings good news about the credibility of registered clinical trials.

Inclusion of children in clinical trials of treatments for COVID-19
This Viewpoint discusses the exclusion of children from coronavirus disease 2019 (COVID-19) clinical trials and why that could harm treatment options for children.

Review evaluates how AI could boost the success of clinical trials
In a review publishing July 17, 2019 in the journal Trends in Pharmacological Sciences, researchers examined how artificial intelligence (AI) could affect drug development in the coming decade.

Kidney patients are neglected in clinical trials
The exclusion of patients with kidney diseases from clinical trials remains an unsolved problem that hinders optimal care of these patients.

Clinical trials beginning for possible preeclampsia treatment
For over 20 years, a team of researchers at Lund University has worked on developing a drug against preeclampsia -- a serious disorder which annually affects around 9 million pregnant women worldwide and is one of the main causes of death in both mothers and unborn babies.

Underenrollment in clinical trials: Patients not the problem
The authors of the study published this month in the Journal of Clinical Oncology investigated why many cancer clinical trials fail to enroll enough patients.

When designing clinical trials for huntington's disease, first ask the experts
Progress in understanding the genetic mutation responsible for Huntington's disease (HD) and at least some molecular underpinnings of the disease has resulted in a new era of clinical testing of potential treatments.

New ALS therapy in clinical trials
New research led by Washington University School of Medicine in St.

Read More: Clinical Trials News and Clinical Trials Current Events is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to